CA3044896A1 - Modified oligonucleotides for treatment of polycystic kidney disease - Google Patents
Modified oligonucleotides for treatment of polycystic kidney disease Download PDFInfo
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- CA3044896A1 CA3044896A1 CA3044896A CA3044896A CA3044896A1 CA 3044896 A1 CA3044896 A1 CA 3044896A1 CA 3044896 A CA3044896 A CA 3044896A CA 3044896 A CA3044896 A CA 3044896A CA 3044896 A1 CA3044896 A1 CA 3044896A1
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- 230000008685 targeting Effects 0.000 description 1
- 229960005187 telmisartan Drugs 0.000 description 1
- 238000011191 terminal modification Methods 0.000 description 1
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- 125000005309 thioalkoxy group Chemical group 0.000 description 1
- 229960003873 thymostimulin Drugs 0.000 description 1
- 230000002916 thymostimulin Effects 0.000 description 1
- 230000000699 topical effect Effects 0.000 description 1
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- 231100000759 toxicological effect Toxicity 0.000 description 1
- 239000012096 transfection reagent Substances 0.000 description 1
- 230000032258 transport Effects 0.000 description 1
- 150000003626 triacylglycerols Chemical class 0.000 description 1
- 125000000169 tricyclic heterocycle group Chemical group 0.000 description 1
- 125000000876 trifluoromethoxy group Chemical group FC(F)(F)O* 0.000 description 1
- 208000037999 tubulointerstitial fibrosis Diseases 0.000 description 1
- 229940035893 uracil Drugs 0.000 description 1
- 230000004143 urea cycle Effects 0.000 description 1
- 229960004699 valsartan Drugs 0.000 description 1
- SJSNUMAYCRRIOM-QFIPXVFZSA-N valsartan Chemical compound C1=CC(CN(C(=O)CCCC)[C@@H](C(C)C)C(O)=O)=CC=C1C1=CC=CC=C1C1=NN=N[N]1 SJSNUMAYCRRIOM-QFIPXVFZSA-N 0.000 description 1
- 210000003462 vein Anatomy 0.000 description 1
- 239000011782 vitamin Substances 0.000 description 1
- 229940088594 vitamin Drugs 0.000 description 1
- 229930003231 vitamin Natural products 0.000 description 1
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- 238000011816 wild-type C57Bl6 mouse Methods 0.000 description 1
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- 229910052725 zinc Inorganic materials 0.000 description 1
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Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Urology & Nephrology (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430139P | 2016-12-05 | 2016-12-05 | |
| US62/430,139 | 2016-12-05 | ||
| PCT/US2017/064428 WO2018106566A1 (en) | 2016-12-05 | 2017-12-04 | Modified oligonucleotides for treatment of polycystic kidney disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3044896A1 true CA3044896A1 (en) | 2018-06-14 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3044896A Pending CA3044896A1 (en) | 2016-12-05 | 2017-12-04 | Modified oligonucleotides for treatment of polycystic kidney disease |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US20200165606A1 (ru) |
| EP (1) | EP3548503A1 (ru) |
| JP (3) | JP7133553B2 (ru) |
| KR (1) | KR20190085951A (ru) |
| CN (1) | CN110036019A (ru) |
| AU (1) | AU2017370560C1 (ru) |
| BR (1) | BR112019011164A2 (ru) |
| CA (1) | CA3044896A1 (ru) |
| CL (1) | CL2019001522A1 (ru) |
| CO (1) | CO2019006234A2 (ru) |
| EA (1) | EA201991360A1 (ru) |
| IL (1) | IL266871A (ru) |
| MA (1) | MA46999A (ru) |
| MX (1) | MX2019006332A (ru) |
| PH (1) | PH12019501224A1 (ru) |
| TW (2) | TW202300647A (ru) |
| WO (1) | WO2018106566A1 (ru) |
| ZA (1) | ZA201903605B (ru) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA44836A (fr) | 2015-08-26 | 2018-07-04 | Univ Texas | Procédés de traitement du syndrome polykystique des reins |
| US11261440B2 (en) | 2017-02-21 | 2022-03-01 | Osaka University | Antisense oligonucleic acid |
| WO2020038968A1 (en) | 2018-08-23 | 2020-02-27 | Roche Innovation Center Copenhagen A/S | Microrna-134 biomarker |
| CN112996568A (zh) * | 2018-11-13 | 2021-06-18 | 莱古路斯治疗法股份有限公司 | 用于调节mir-10b活性的微小rna化合物和方法 |
| CN118103510A (zh) * | 2021-10-08 | 2024-05-28 | 莱古路斯治疗法股份有限公司 | 用于治疗多囊性肾病的方法和组合物 |
| WO2024196998A1 (en) | 2023-03-22 | 2024-09-26 | Regulus Therapeutics, Inc. | Methods for treating nervous system disorders |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2848238C (en) | 2006-10-03 | 2016-07-19 | Tekmira Pharmaceuticals Corporation | Lipid containing formulations |
| US8288356B2 (en) | 2007-10-04 | 2012-10-16 | Santaris Pharma A/S | MicroRNAs |
| AU2009221064B2 (en) * | 2008-03-07 | 2014-12-11 | Roche Innovation Center Copenhagen A/S | Pharmaceutical compositions for treatment of microRNA related diseases |
| CA2780726A1 (en) * | 2009-11-11 | 2011-05-19 | Tariq M. Rana | Method for generation and regulation of ips cells and compositions thereof |
| US20160362688A1 (en) * | 2014-02-12 | 2016-12-15 | Thomas Jefferson University | Compositions and methods of using microrna inhibitors |
| MA44836A (fr) * | 2015-08-26 | 2018-07-04 | Univ Texas | Procédés de traitement du syndrome polykystique des reins |
-
2017
- 2017-12-04 BR BR112019011164A patent/BR112019011164A2/pt unknown
- 2017-12-04 CA CA3044896A patent/CA3044896A1/en active Pending
- 2017-12-04 EA EA201991360A patent/EA201991360A1/ru unknown
- 2017-12-04 CN CN201780074216.4A patent/CN110036019A/zh active Pending
- 2017-12-04 MA MA046999A patent/MA46999A/fr unknown
- 2017-12-04 AU AU2017370560A patent/AU2017370560C1/en active Active
- 2017-12-04 JP JP2019529933A patent/JP7133553B2/ja active Active
- 2017-12-04 TW TW111120773A patent/TW202300647A/zh unknown
- 2017-12-04 MX MX2019006332A patent/MX2019006332A/es unknown
- 2017-12-04 TW TW106142317A patent/TWI769197B/zh active
- 2017-12-04 KR KR1020197016357A patent/KR20190085951A/ko not_active Application Discontinuation
- 2017-12-04 US US16/463,041 patent/US20200165606A1/en not_active Abandoned
- 2017-12-04 WO PCT/US2017/064428 patent/WO2018106566A1/en active Application Filing
- 2017-12-04 EP EP17823261.7A patent/EP3548503A1/en active Pending
-
2019
- 2019-05-26 IL IL266871A patent/IL266871A/en unknown
- 2019-05-31 PH PH12019501224A patent/PH12019501224A1/en unknown
- 2019-06-04 CL CL2019001522A patent/CL2019001522A1/es unknown
- 2019-06-05 ZA ZA2019/03605A patent/ZA201903605B/en unknown
- 2019-06-14 CO CONC2019/0006234A patent/CO2019006234A2/es unknown
-
2020
- 2020-09-25 US US16/948,620 patent/US20210095282A1/en not_active Abandoned
-
2022
- 2022-07-25 US US17/814,647 patent/US20230109466A1/en active Pending
- 2022-08-29 JP JP2022135567A patent/JP2022169726A/ja active Pending
-
2024
- 2024-06-24 JP JP2024101134A patent/JP2024123177A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7133553B2 (ja) | 2022-09-08 |
| PH12019501224A1 (en) | 2019-09-23 |
| US20200165606A1 (en) | 2020-05-28 |
| AU2017370560B2 (en) | 2021-11-18 |
| JP2019536804A (ja) | 2019-12-19 |
| CN110036019A (zh) | 2019-07-19 |
| TWI769197B (zh) | 2022-07-01 |
| US20230109466A1 (en) | 2023-04-06 |
| EP3548503A1 (en) | 2019-10-09 |
| TW201821618A (zh) | 2018-06-16 |
| AU2017370560C1 (en) | 2022-08-11 |
| MX2019006332A (es) | 2019-08-01 |
| TW202300647A (zh) | 2023-01-01 |
| IL266871A (en) | 2019-07-31 |
| ZA201903605B (en) | 2023-12-20 |
| CL2019001522A1 (es) | 2019-10-25 |
| KR20190085951A (ko) | 2019-07-19 |
| US20210095282A1 (en) | 2021-04-01 |
| EA201991360A1 (ru) | 2019-11-29 |
| BR112019011164A2 (pt) | 2019-10-08 |
| JP2024123177A (ja) | 2024-09-10 |
| CO2019006234A2 (es) | 2019-08-30 |
| AU2017370560A1 (en) | 2019-06-06 |
| WO2018106566A1 (en) | 2018-06-14 |
| JP2022169726A (ja) | 2022-11-09 |
| MA46999A (fr) | 2019-10-09 |
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